Ultragenyx's Angelman Syndrome Drug Fails in Critical Trial
By Editor • September 2, 2026 • 1 min read
Ultragenyx announced on Wednesday that its drug GTX-102, designed to treat Angelman syndrome, failed to show any significant benefit over a placebo in a Phase 3 trial.
This disappointing outcome follows earlier promising results that had sparked hope among families affected by the disorder. The company, which has other approved treatments for ultra-rare diseases, had pinned hopes for profitability on this drug.
Source: www.statnews.com
#Angelman syndrome #biotech #clinical trial #GTX-102 #Ultragenyx
